United Therapeutics (UTHR) investor relations material

Study design and population

• Phase 3 TETON-2 was a randomized, placebo-controlled trial evaluating inhaled treprostinil in patients with idiopathic pulmonary fibrosis (IPF), with 298 in the treatment group and 295 in placebo.

• Primary endpoint was change in absolute forced vital capacity (FVC) from baseline to week 52; secondary endpoints included time to clinical worsening, acute exacerbation, overall survival, percent predicted FVC, KBILD, and DLCO.

• Key inclusion criteria: age 40+, FVC ≥45% predicted, confirmed IPF diagnosis, and stable background therapy if applicable.

• Baseline characteristics were balanced between groups, with mean age ~72 years and similar distribution of background therapies.

Efficacy results

• The primary endpoint was met: inhaled treprostinil showed a placebo-corrected median treatment effect of 95.6 mL improvement in FVC at week 52 (95% CI, 52.2–139.0; P<0.0001).

• Statistically significant improvements were observed in several secondary endpoints, including time to first clinical worsening (HR 0.71, P=0.0190), percent predicted FVC (2.7% difference, P<0.0001), KBILD (2.29 point difference, P=0.0124), and DLCO (1.91% difference, P=0.0416).

• Subgroup analyses showed consistent FVC benefit across background therapies (nintedanib, pirfenidone, or none) and demographic groups.

Safety and tolerability

• Inhaled treprostinil was well tolerated, with a safety profile consistent with previous studies and known prostacyclin-related adverse events.

• Most common adverse events included cough (48.3%), headache (24.1%), and diarrhea (19.8%).

• Serious adverse events and deaths were similar between groups; 3.4% of patients in the treatment group had AEs leading to death versus 2.0% in placebo.